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A Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions

Primary Purpose

CVI - Common Variable Immunodeficiency

Status

Completed

Phase

Phase 4

Locations

United States

Study Type

Interventional

Intervention

C1-esterase inhibitor [recombinant] (C1-INH-R)

About this trial

This is an interventional prevention trial for CVI - Common Variable Immunodeficiency focused on measuring Intravenous Immunoglobulin, C1-esterase inhibitor, Adverse drug reaction (ADR)

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Age 18 years and older experiencing ADRs related to IVIG infusions
Stable dose of IVIG for 3 months
Willing to comply with all aspects of the protocol, including blood draws
Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception. Acceptable forms of contraception are defined as those with a failure rate < 1% when properly applied and include: a combination oral pill, some intra-uterine devices, and a sterilized partner in a stable relationship. Female patients must not be pregnant, planning to become pregnant, or be actively breastfeeding through the entire study period.

Exclusion Criteria:

Receiving treatment for HAE, either prophylactic or acute therapy
Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant.
Patients who, in the investigator's opinion, might not be suitable for the trial

Sites / Locations

IMMUNOe Research Centers

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

C1-esterase inhibitor [recombinant] (C1-INH-R)

Arm Description

Single-site, open-label arm to evaluate the benefit of C1-INH-R in subjects on IVIG therapy who experience ADRs. The study will have 2 periods: 6 - 8 weeks - subjects will receive 2 infusions of IVIG 9 - 12 weeks - subjects will receive 3 infusions of C1-INH-R prior to IVIG infusion

Outcomes

Primary Outcome Measures

The change in Modified Fatigue Severity Scale (mFSS)

Subject-rated Rasch-built 7-item modified fatigue survey scale. Scale rated 0 - 3 (3 = agree, 0 = less agree)

The change in Modified Fatigue Impact Scale (MFIS)

Subject-rated 24 item questionnaire measuring the impact of fatigue rated 0 - 4 (0 = never, 1 = rarely, 2 = sometimes, 3 = often, 4 = almost always)

The change in Migraine Disability Assessment (MIDAS)

Headache severity measurement of number of days affected after infusion

The change in Headache Impact Scale (HIT-6)

Subject-rated measurement of the impact headaches have on the ability to function on the job, at school, at home and in social situations. 6 item questionnaire rated from never (6 points each), rarely (8 points each), sometimes (10 points each), very often (11 points each), always (13 points each). The higher number the more the impact.

The change in Activities of Daily Living Sliding Scale

Subject-rated 10 point scale measuring level of activity with 1 being the worst (least) and 10 being the best (greatest)

The change in Activities of Daily Living Questionnaire

Calculates the number of days missed from work, school/daycare/activities, housework, and regular exercise

The change in Energy Sliding Scale

Subject-rated10 point scale measuring energy level with 1 being the worst (Lowest) and 10 being the best (highest)

The change in Infection Questionnaire

Subject-rated 7 item infection questionnaire measured from 1 to 10 with 1 being the least affected and 10 being the most affected.

The change in Perceived Deficits Questionnaire - cognitive assessment

Subject-rated 20 item questionnaire measuring memory, attention and concentration rated 0 to 4 (0 = never, 1 = rarely, 2 = sometimes, 3 = often, 4 = almost always) with the lower score being least impact.

The change in 36 item short form survey (SF-36)

Health Survey asks 36 questions to measure functional health and well-being from the patient's point of view.

Change in the number of ADRs

Adverse reactions to infusions

Secondary Outcome Measures

Change in levels of C1-INH pre- and post-infusion

Laboratory levels of C1-INH total and functional

Full Information

NCT ID

NCT03576469

First Posted

June 15, 2018

Last Updated

February 11, 2021

Sponsor

IMMUNOe Research Centers

1. Study Identification

Unique Protocol Identification Number

NCT03576469

Brief Title

A Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions

Official Title

A Single-site, Open-Label, Pilot Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions

Study Type

Interventional

2. Study Status

Record Verification Date

February 2021

Overall Recruitment Status

Completed

Study Start Date

June 13, 2018 (Actual)

Primary Completion Date

December 1, 2019 (Actual)

Study Completion Date

March 1, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

IMMUNOe Research Centers

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

Patients receiving intravenous immunoglobulin (IVIG) therapy for primary immunodeficiency and neurologic conditions may experience adverse drug reactions (ADRs). The mechanism of the ADR is unknown. Currently, the standard practice for these patients is to change from IV to subcutaneous IG (SCIG) but because of the need of immunomodulation or patient preference, SCIG may not be an option. Data has shown that some levels of complement decrease from pre- to post-infusion of IVIG. This study is to determine if replacing this complement protein may ameliorate ADRs.

Detailed Description

This is a single-site, pilot study conducted in the US to determine the benefit of human C1-esterase inhibitor [recombinant] (C1-INH-R) therapy to ameliorate ADRs in subjects receiving IVIG therapy experience ADRs post-infusion. Subjects who are currently receiving IVIG for immunodeficiency or neurologic conditions and experience ADRs will be enrolled. In part 1, subjects will continue to receive IVIG for two infusions. Complement proteins will be measured pre- and post-infusion. Quality of life and other questionnaires will be administered. In part 2, subjects will receive C1-INH-R infusion prior to receiving IVIG infusion for three infusions. Complement proteins will be measured pre- and post- infusion. Quality of life and other questionnaires will be administered.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

CVI - Common Variable Immunodeficiency

Keywords

Intravenous Immunoglobulin, C1-esterase inhibitor, Adverse drug reaction (ADR)

7. Study Design

Primary Purpose

Prevention

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Model Description

Single Group

Masking

None (Open Label)

Allocation

N/A

Enrollment

20 (Actual)

8. Arms, Groups, and Interventions

Arm Title

C1-esterase inhibitor [recombinant] (C1-INH-R)

Arm Type

Experimental

Arm Description

Intervention Type

Biological

Intervention Name(s)

C1-esterase inhibitor [recombinant] (C1-INH-R)

Intervention Description

C1-INH-R is FDA approved and indicated for the treatment of acute attacks of angioedema in adolescent and adult patients with Hereditary Angioedema (HAE) as a replacement for low levels of C1-esterase inhibitor or low function of C1-esterase inhibitor

Primary Outcome Measure Information:

Title

The change in Modified Fatigue Severity Scale (mFSS)

Description

Subject-rated Rasch-built 7-item modified fatigue survey scale. Scale rated 0 - 3 (3 = agree, 0 = less agree)

Time Frame